Nevertheless, SMN upregulation protects against mutant SOD1-induced cell death in an immortalized motor neuronal cell line (NSC-34) (Zou et al., 2007), rescues mutant FUS-mediated axonal defects in primary cortical neurons (Groen et al., 2013), and can improve survival of iPSC-derived motor neurons differentiated from SOD1 and TDP-43 ALS patient fibroblasts (Rodriguez-Muela et al., 2017). The gene discussed is SMN2; the disease is amyotrophic lateral sclerosis.